Sangamo, Novartis Partner to Develop Gene Therapies for Autism

Sangamo Therapeutics, Inc. has entered a global licensing collaboration agreement with Novartis to develop and commercialize gene regulation therapies to address three neurodevelopmental targets, including autism spectrum disorder (ASD) and other neurodevelopmental disorders. The collaboration will leverage Sangamo’s genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), to aim to upregulate the expression of key genes involved in neurodevelopmental disorders.

Novartis will pay $75 million upfront and Sangamo is eligible to earn up to $720 million in other development and commercial milestone payments. Sangamo is also eligible to receive from royalties on potential sales of products arising from the collaboration.

Sangamo’s ZFP-TF genome regulation technology, which is currently delivered with adeno-associated viruses (AAVs), functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. The collaboration will leverage ZFP-TFs engineered by Sangamo in an effort to upregulate, or activate, the expression of genes that are inadequately expressed in individuals with certain types of neurodevelopmental disorders.

Over a three-year collaboration period, Novartis has exclusive rights to ZFP-TFs targeted to three undisclosed genes associated with neurodevelopmental disorders, including ASD and intellectual disability. Novartis also has the option to license Sangamo’s proprietary AAVs. Sangamo is responsible for certain research and associated manufacturing activities, which will be funded by Novartis, and Novartis assumes responsibility for additional research activities, clinical development, regulatory interactions, manufacturing and global commercialization.